2019 ANNUAL REPORT amfAR, The Foundation for AIDS Research Cover photo: Human cells. 3D illustration © Ustyna Shevhcuk | Dreamstime.com Contents amfAR in 2019 01 Grants, Fellowships, and Awards 09 Research Grants, Fellowships, and Awards TREAT Asia Grants and Awards Public Policy Awards Financial Highlights 15 Leadership and Advisory Committees 17 Board of Trustees Scientific Advisory Committee Program Advisory Council Management Group Our Mission: amfAR, The Foundation for AIDS Research, is dedicated to ending the global AIDS epidemic through innovative research. HIGHLIGHTS RESEARCH edge technology to address the main barrier to an HIV cure: the persistent reservoirs of virus not cleared by antiretroviral therapy (ART). Totaling $800,000, this new round of Investment grants launched the critical third phase of a research project begun Countdown to a Cure for AIDS in 2017. In 2015, amfAR launched its Countdown to a Cure for AIDS initiative, which is aimed at developing the scientific basis for a cure. To date, In the first two phases of her study, Dr. Hui Zhang of Johns amfAR has awarded 79 Countdown grants totaling more than $48 Hopkins University used mass spectrometry to scan the surface of million to support research conducted by 277 scientists working human cells for proteins—biomarkers—that discriminate between at 95 institutions in 15 countries. Structured to provide sustained latently infected and uninfected cells. After scanning a variety support for a wide range of studies that advance both emerging and of cell lines, she identified 17 potential targets. In phase three, established ideas, the strategy comprises the following components: Dr. Zhang is teaming up with HIV scientist Dr. Weiming Yang, also of Johns Hopkins University, to determine in a preclinical u The amfAR Institute for HIV Cure Research was established study whether specifically killing cells displaying any of these 17 in 2015 with a $20 million grant over five years to the University proteins will eliminate the latent reservoir. of California, San Francisco (UCSF). Researchers at the Institute have been laying the groundwork for a complex clinical study u Impact Grants of up to $2 million each over four years support employing a three-pronged approach to eradicating HIV. A the in-depth development of concepts already underpinned by therapeutic vaccine will be combined with an immune adjuvant—a preliminary data showing genuine potential for achieving a cure. drug often used to boost the efficacy of vaccines—that has been In September 2018, amfAR awarded grants totaling $828,000 to shown in clinical trials to have additional anti-HIV effects. The advance two critical areas of HIV cure research. Five grants are third component combines two broadly neutralizing antibodies, supporting a range of efforts to understand the mechanisms and the source of much optimism in the cure field for their ability not predictors of post-treatment control, whereby a small number of only to kill viruses that are produced by infected cells, but also to individuals are able to control their HIV after stopping treatment. direct the immune system to kill the cells producing those viruses. Three additional grantees are studying HIV-positive populations in low- and middle-income countries to look for differences in how u Investment Grants, awarded over a four-year period, are the persistent viral reservoir forms and changes over time. aimed at recruiting the experience and expertise of scientists from outside the field of HIV. Grantees may have expertise in u ARCHE, the amfAR Research Consortium on HIV Eradication, fields such as cancer, neuroscience, or inflammatory disease supports collaborative teams of scientists in the U.S. and around that can directly inform efforts to cure HIV. In February 2019, the world working on a range of HIV cure strategies. In September amfAR announced new funding to researchers using cutting- 2018, amfAR awarded $800,000 in new ARCHE funding to six teams of researchers developing an ambitious gene therapy- 1 based approach to curing HIV. The award launched a critical new u The Mathilde Krim Fellowships in Basic Biomedical Research phase in a study initiated in 2017. In a three-pronged attack on the support bright young scientists advancing innovative solutions to HIV reservoir, the researchers are employing broadly neutralizing HIV/AIDS. In September 2018, amfAR announced the recipient of antibodies, CAR stem cells—cells genetically reprogrammed the 2018 Mathilde Krim Fellowship in Basic Biomedical Research: to recognize and attack infected cells—and molecular scissors Dr. Yen-Ting Lai of the Vaccine Research Center/National targeting the virus. The grant was supported in part by the Bill and Institutes of Health in Bethesda, MD. The 2019 Krim fellows, Dr. Melinda Gates Foundation. Maolin Lu of Yale University in New Haven, CT, and Dr. Shaheed Abdulhaqq, of Oregon Health and Science University in Portland, The investigators are: Dr. Hildegard Büning of Hannover Medical OR, were announced in September 2019. Each fellow is awarded School, Germany; Dr. Keith Jerome of the University of Washington, approximately $150,000 over two years. Seattle; Dr. Hans-Peter Kiem of Fred Hutchinson Cancer Research Center, Seattle; Dr. Scott Kitchen of UCLA; Dr. Yasuhiro Takeuchi of University College London; Dr. Drew Weissman of University of Pennsylvania in Philadelphia; and Dr. Richard Wyatt of The Scripps Research Institute in La Jolla, CA. In June 2019, amfAR awarded $1.16 million in ARCHE grants for a pair of innovative research studies pursuing different angles to attack HIV. Dr. Keith Jerome of the University of Washington, Seattle, was awarded $344,000 for a project that aims to advance a cost-effective in vivo gene therapy strategy for curing HIV in which gene-editing tools are injected directly into the patient using vectors that function like ‘Trojan horses.’ And Dr. Jonathan Li of Brigham and Women’s Hospital, Boston, was awarded $815,000 for a project studying post-treatment control of the virus. Dr. Li and his team are gathering and analyzing samples from a multinational cohort of post-treatment controllers Dr. Shaheed Abdulhaqq to investigate whether characteristics of the virus or immunologic responses predict control. Discovering what leads to post-treatment Published Research control in some people could help to achieve durable ART-free control in all people living with HIV. Research studies make the greatest impact on the HIV field and on the broader scientific community when they are published in scientific journals. In FY2019, 53 scientific publications resulted from amfAR-funded research. The “London Patient” In the March 2019 issue of Nature, researchers affiliated with amfAR’s IciStem consortium reported on the “London patient,” who may be only the second person to have been cured of HIV following a stem cell transplant. This case—along with the “Düsseldorf patient,” who may also have been cured—is providing critical new data to compare with the case of the “Berlin patient,” the first known case of a cure. A Family Trait CCR5, the primary receptor enabling HIV to infect cells, is known for its role in the cure of the “Berlin patient” following a stem cell transplant with donor cells bearing a mutated CCR5 protein. In the April 2019 issue of eLIFE, Dr. Steven Deeks of UCSF and colleagues reported that people with a natural ability to control their HIV without ART had less CCR5 present on their cells than noncontrollers, and their cells were less permissive to HIV infection. Furthermore, natural resistance to HIV through reduced levels of CCR5 was shown to be inherited. Dr. Yen-Ting Lai 2 Identifying a Biomarker for the ‘Active’ HIV Reservoir PUBLIC POLICY In the May 2019 issue of Pathogens and Immunity, Drs. Timothy Henrich and Peter Hunt, both at UCSF, reported on a potential biomarker of the “active reservoir”—persistent virus that is probably Informed by thorough research and analysis, amfAR is a highly first to re-seed infection when ART is stopped. Thinking on the respected advocate of rational and compassionate HIV/AIDS- nature of the reservoir is evolving to include not only latent, but also related public policy. The Foundation is engaged in efforts to active reservoir. Differentiating cells harboring active reservoir from secure necessary increases in funding for HIV/AIDS research and healthy, uninfected cells would be an important advance. global HIV/AIDS programs, expand access to treatment and care for marginalized populations, advocate harm reduction policies aimed at reducing the spread of HIV and hepatitis C (HCV) among HIV Cure Summit people who inject drugs, and protect the civil rights of all people In December 2018, amfAR held its fifth annual HIV Cure Summit at affected by or vulnerable to HIV/AIDS. UCSF, where the amfAR Institute for HIV Cure Research is based. Leading amfAR-funded cure researchers detailed their progress Ending the Domestic HIV Epidemic and discussed the scientific challenges that stand in the way of a cure. Dr. Rowena Johnston, amfAR vice president and director In 2019, President Trump announced a plan to end HIV of research, elucidated the two types of cure that researchers are transmissions in the U.S. by 2030, targeting geographic hotspots pursuing: eradication and post-treatment control. In eradication, an of infection. The Ending the HIV Epidemic (EHE) initiative aims individual completely clears HIV from his or her body, while in post- to reduce HIV infections by 75% in five years, and by 90% in treatment control some virus remains but the immune system can ten years. In 2019, amfAR launched a free interactive database control it without ART. Dr. Steven Deeks, a professor of medicine at (ehe.amfar.org) to help policymakers, public health officials, UCSF and a lead investigator at the amfAR Institute, explained the advocates, and other stakeholders understand the opportunities complex clinical study he is about to embark on.